首页 > 最新文献

Value in Health最新文献

英文 中文
Does Real Option Value Influence Oncologists' Treatment Recommendations? A Survey of US Oncologists. 实际期权价值会影响肿瘤学家的治疗建议吗?对美国肿瘤学家的调查。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-09 DOI: 10.1016/j.jval.2024.07.021
Meng Li, Zizi Elsisi, William Wong, Stacey Kowal, David L Veenstra, Louis P Garrison

Objectives: Survival benefit from anticancer treatments, even if modest, improves a patient's chances of accessing future innovations, thereby creating real option value. There is no empirical evidence on the impact of potential future innovations on oncologists' treatment recommendations.

Methods: We conducted a national online survey of practicing medical and hematological oncologists. We presented a hypothetical metastatic cancer patient with median survival of 6 months under 4 decision-making scenarios with varying expected efficacy and time to arrival of future innovations. We assessed the likelihood of discussing future innovations with their patients and the likelihood that future innovations would influence their current treatment recommendation, as well as factors associated with these 2 outcomes using multivariate logistic regressions.

Results: A total of 201 oncologists completed the survey. When future innovations were expected to improve survival by 6 months and be available in 6 months, 76% of oncologists were likely or very likely to discuss the innovations with their patients, and 68% reported they would influence their current treatment recommendations. A 1-month increase in the expected survival improvement of future innovation was associated with a 1.17 greater odds (95% CI 1.1-1.25) of reporting likely or very likely to discuss future innovations with their patients, whereas a 1-month increase in the expected time to arrival was associated with a 0.91 lower odds (95% CI 0.88-0.94).

Conclusions: Given that potential future innovations seem to influence oncologists' treatments recommendations, evidence to inform clinical guidelines and value assessments should consider data on real option value impacts to support informed treatment decision making.

目标:抗癌治疗带来的生存获益,即使微不足道,也能提高患者获得未来创新治疗的机会,从而创造实际期权价值(ROV)。关于未来潜在创新对肿瘤学家治疗建议的影响,目前尚无实证证据:我们对执业的内科和血液肿瘤专家进行了一次全国性在线调查。我们假设一名中位生存期为 6 个月的转移性癌症患者在四种决策情景下接受治疗,这些情景的预期疗效和未来创新的到来时间各不相同。我们使用多变量逻辑回归评估了与患者讨论未来创新的可能性和未来创新影响其当前治疗建议的可能性,以及与这两种结果相关的因素。当未来的创新技术有望在6个月内提高生存率并在6个月内上市时,76%的肿瘤学家可能或非常可能与患者讨论这些创新技术,68%的肿瘤学家表示这些创新技术将影响他们目前的治疗建议。未来创新疗法的预期生存期改善率每增加一个月,肿瘤专家表示可能或非常可能与患者讨论未来创新疗法的几率就会增加1.17(95% CI:1.1-1.25),而预期到达时间每增加一个月,几率就会降低0.91(95% CI:0.88-0.94):由于潜在的未来创新似乎会影响肿瘤学家的治疗建议,因此临床指南和价值评估的参考证据应考虑有关ROV影响的数据,以支持知情的治疗决策。
{"title":"Does Real Option Value Influence Oncologists' Treatment Recommendations? A Survey of US Oncologists.","authors":"Meng Li, Zizi Elsisi, William Wong, Stacey Kowal, David L Veenstra, Louis P Garrison","doi":"10.1016/j.jval.2024.07.021","DOIUrl":"10.1016/j.jval.2024.07.021","url":null,"abstract":"<p><strong>Objectives: </strong>Survival benefit from anticancer treatments, even if modest, improves a patient's chances of accessing future innovations, thereby creating real option value. There is no empirical evidence on the impact of potential future innovations on oncologists' treatment recommendations.</p><p><strong>Methods: </strong>We conducted a national online survey of practicing medical and hematological oncologists. We presented a hypothetical metastatic cancer patient with median survival of 6 months under 4 decision-making scenarios with varying expected efficacy and time to arrival of future innovations. We assessed the likelihood of discussing future innovations with their patients and the likelihood that future innovations would influence their current treatment recommendation, as well as factors associated with these 2 outcomes using multivariate logistic regressions.</p><p><strong>Results: </strong>A total of 201 oncologists completed the survey. When future innovations were expected to improve survival by 6 months and be available in 6 months, 76% of oncologists were likely or very likely to discuss the innovations with their patients, and 68% reported they would influence their current treatment recommendations. A 1-month increase in the expected survival improvement of future innovation was associated with a 1.17 greater odds (95% CI 1.1-1.25) of reporting likely or very likely to discuss future innovations with their patients, whereas a 1-month increase in the expected time to arrival was associated with a 0.91 lower odds (95% CI 0.88-0.94).</p><p><strong>Conclusions: </strong>Given that potential future innovations seem to influence oncologists' treatments recommendations, evidence to inform clinical guidelines and value assessments should consider data on real option value impacts to support informed treatment decision making.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914158","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cost-of/Burden-of-Illness Studies: Steps Backward? 疾病成本/负担研究:退步?
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-09 DOI: 10.1016/j.jval.2024.07.023
{"title":"Cost-of/Burden-of-Illness Studies: Steps Backward?","authors":"","doi":"10.1016/j.jval.2024.07.023","DOIUrl":"10.1016/j.jval.2024.07.023","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914155","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Cost-Effectiveness of Seizure Dogs for Persons Living With Severe Refractory Epilepsy: Results From the EPISODE Study. 严重难治性癫痫患者使用癫痫犬的成本效益:EPISODE 研究的结果。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-09 DOI: 10.1016/j.jval.2024.07.020
Valérie van Hezik-Wester, Saskia de Groot, Tim Kanters, Louis Wagner, Jacqueline Ardesch, Werner Brouwer, Isaac Corro-Ramos, Job van Exel, Matthijs Versteegh

Objectives: The Epilepsy Support Dog Evaluation study was commissioned by the Dutch Ministry of Health, Welfare and Sports to inform a reimbursement decision on seizure dogs. The randomized trial found that seizure dogs reduce seizure frequency and improve health-related quality of life of persons with severe refractory epilepsy (PSREs). This article examined the cost-effectiveness (CE) of adding seizure dogs to usual care for PSREs in The Netherlands.

Methods: A microsimulation model was developed, informed by generalized linear mixed models using patient-level trial data from the Epilepsy Support Dog Evaluation study. The model adopted a 10-year time horizon and took a societal perspective. Seizure frequency was predicted as a function of time with the seizure dog. Patient utilities, caregiver utilities, and costs were predicted as a function of seizure frequency and time with the seizure dog.

Results: Quality-adjusted life-years (QALYs) of PSREs with a seizure dog and usual care alone were estimated at 6.28 and 5.65, respectively (Δ 0.63). For caregivers, estimated QALYs were 6.94 and 6.52, respectively (Δ 0.42). Total costs were respectively €228 691 and €226 261 (Δ €2430). Intervention costs were largely offset by savings in informal care and healthcare. The incremental CE ratio was €2314/QALY. Probabilistic sensitivity analysis indicated a 91% probability of seizure dogs being cost-effective at the €50 000/QALY threshold. The incremental CE ratio fell well below this threshold in scenario analyses.

Conclusions: Seizure dogs are likely to be a cost-effective addition to usual care for PSREs in The Netherlands.

研究目的EPISODE 研究由荷兰卫生、福利和体育部委托进行,旨在为癫痫发作犬的报销决定提供参考。随机试验发现,癫痫发作犬可降低严重难治性癫痫患者(PSREs)的癫痫发作频率,并改善其与健康相关的生活质量。本文探讨了在荷兰为 PSREs 患者提供常规护理的基础上添加癫痫犬的成本效益:方法:利用 EPISODE 研究中患者层面的试验数据,通过广义线性混合模型建立了一个微观模拟模型。该模型的时间跨度为 10 年,并采用了社会视角。癫痫发作频率是通过使用癫痫发作犬的时间来预测的。患者的效用、护理人员的效用和成本则作为癫痫发作频率和使用癫痫犬时间的函数进行预测:使用癫痫犬进行 PSRE 和仅使用常规护理的质量调整生命年 (QALY) 估计分别为 6.28 和 5.65(Δ 0.63)。对于护理人员,估计的QALY分别为6.94和6.52(Δ 0.42)。总成本分别为 228,691 欧元和 226,261 欧元(Δ 2,430 欧元)。干预成本在很大程度上被非正式护理和医疗保健方面节省的费用所抵消。增量成本效益比(ICER)为 2314 欧元/QALY。概率敏感性分析表明,在 50,000 欧元/QALY 临界值时,癫痫犬具有成本效益的概率为 91%。在情景分析中,ICER 远远低于这一阈值:在荷兰,癫痫犬很可能是对 PSRE 常规护理的一种具有成本效益的补充。
{"title":"The Cost-Effectiveness of Seizure Dogs for Persons Living With Severe Refractory Epilepsy: Results From the EPISODE Study.","authors":"Valérie van Hezik-Wester, Saskia de Groot, Tim Kanters, Louis Wagner, Jacqueline Ardesch, Werner Brouwer, Isaac Corro-Ramos, Job van Exel, Matthijs Versteegh","doi":"10.1016/j.jval.2024.07.020","DOIUrl":"10.1016/j.jval.2024.07.020","url":null,"abstract":"<p><strong>Objectives: </strong>The Epilepsy Support Dog Evaluation study was commissioned by the Dutch Ministry of Health, Welfare and Sports to inform a reimbursement decision on seizure dogs. The randomized trial found that seizure dogs reduce seizure frequency and improve health-related quality of life of persons with severe refractory epilepsy (PSREs). This article examined the cost-effectiveness (CE) of adding seizure dogs to usual care for PSREs in The Netherlands.</p><p><strong>Methods: </strong>A microsimulation model was developed, informed by generalized linear mixed models using patient-level trial data from the Epilepsy Support Dog Evaluation study. The model adopted a 10-year time horizon and took a societal perspective. Seizure frequency was predicted as a function of time with the seizure dog. Patient utilities, caregiver utilities, and costs were predicted as a function of seizure frequency and time with the seizure dog.</p><p><strong>Results: </strong>Quality-adjusted life-years (QALYs) of PSREs with a seizure dog and usual care alone were estimated at 6.28 and 5.65, respectively (Δ 0.63). For caregivers, estimated QALYs were 6.94 and 6.52, respectively (Δ 0.42). Total costs were respectively €228 691 and €226 261 (Δ €2430). Intervention costs were largely offset by savings in informal care and healthcare. The incremental CE ratio was €2314/QALY. Probabilistic sensitivity analysis indicated a 91% probability of seizure dogs being cost-effective at the €50 000/QALY threshold. The incremental CE ratio fell well below this threshold in scenario analyses.</p><p><strong>Conclusions: </strong>Seizure dogs are likely to be a cost-effective addition to usual care for PSREs in The Netherlands.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914160","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Direct and Indirect Mapping of Assessment of Quality of Life - 6 Dimensions (AQoL-6D) Onto EQ-5D-5L Utilities Using Data From a Multicenter, Cross-Sectional Study of Malaysians With Chronic Heart Failure. 利用马来西亚慢性心力衰竭患者的多中心横断面研究数据,将 AQoL-6D 直接和间接映射到 EQ-5D-5L 工具。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-09 DOI: 10.1016/j.jval.2024.07.016
Yi Jing Tan, Siew Chin Ong

Objectives: The Assessment of Quality of Life - 6 Dimensions (AQoL-6D), a generic preference-based measure, is an appealing alternative to EQ-5D-5L for assessing health status in patients with chronic heart failure (HF), given its expanded scope. However, without a Malaysian value set, the AQoL-6D cannot generate health state utility values (HSUVs) to support local economic evaluations. This study intended to develop algorithms for predicting EQ-5D-5L HSUVs from AQoL-6D in an HF population.

Methods: Cross-sectional data from a multicenter cohort of 419 HF outpatients were used. Both direct and indirect mapping approaches were attempted using 5 sets of explanatory variables and 8 models (ordinary least squares, Tobit, censored least absolute deviations, generalized linear model, 2-part model [TPM], beta regression-based model, adjusted limited dependent variable mixture model, and multinomial ordinal regression [MLOGIT]). The models' predictive performance was assessed through 10-fold cross-validated mean absolute error [MAE] and root mean squared error [RMSE]). Potential prediction bias was also examined graphically. The best-performing models, with the lowest RMSE and no bias, were then identified.

Results: Among the models evaluated, TPM, which included age, sex, and 5 AQoL-6D dimension scores as predictors, appears to be the best-performing model for directly predicting EQ-5D-5L HSUVs from AQoL-6D. TPM yielded the lowest MAE (0.0802) and RMSE (0.1116), and demonstrated predictive accuracy for HSUVs >0.2 without significant bias. A MLOGIT model developed for response mapping had suboptimal predictive accuracy.

Conclusions: This study developed potentially useful mapping algorithms for generating Malaysian EQ-5D-5L HSUVs from AQoL-6D responses among patients with HF when direct EQ-5D-5L data are unavailable.

目的:AQoL-6D 是一种基于偏好的通用测量方法,由于其测量范围更广,因此是评估慢性心力衰竭(HF)患者健康状况的 EQ-5D-5L 的替代方法,很有吸引力。然而,由于没有马来西亚的价值集,AQoL-6D 无法生成健康状态效用值 (HSUV) 以支持当地的经济评估。本研究旨在开发算法,根据 AQoL-6D 预测高频人群的 EQ-5D-5L HSUV:方法:研究使用了多中心队列中 419 名高频门诊患者的横断面数据。使用五组解释变量和 8 个模型(普通最小二乘法、托比特模型、删减最小绝对偏差模型、广义线性模型、两部分模型 [TPM]、基于贝塔回归的模型、调整有限因变量混合模型和多项式序数回归 [MLOGIT])尝试了直接和间接映射方法。模型的预测性能通过十倍交叉验证的平均绝对误差(MAE)和均方根误差(RMSE)进行评估。)此外,还对潜在的预测偏差进行了图形分析。然后确定了 RMSE 最低且无偏差的最佳模型:结果:在所评估的模型中,包括年龄、性别和五个 AQoL-6D 维度分数作为预测因子的 TPM 似乎是通过 AQoL-6D 直接预测 EQ-5D-5L HSUV 的最佳模型。TPM 的 MAE(0.0802)和 RMSE(0.1116)均最低,对 HSUV >0.2 的预测准确性也很高,且无明显偏差。为反应映射开发的 MLOGIT 模型的预测准确性不理想:本研究开发了一种潜在有用的映射算法,可在无法获得直接 EQ-5D-5L 数据的情况下,根据 HF 患者的 AQoL-6D 反应生成马来西亚 EQ-5D-5L HSUV。
{"title":"Direct and Indirect Mapping of Assessment of Quality of Life - 6 Dimensions (AQoL-6D) Onto EQ-5D-5L Utilities Using Data From a Multicenter, Cross-Sectional Study of Malaysians With Chronic Heart Failure.","authors":"Yi Jing Tan, Siew Chin Ong","doi":"10.1016/j.jval.2024.07.016","DOIUrl":"10.1016/j.jval.2024.07.016","url":null,"abstract":"<p><strong>Objectives: </strong>The Assessment of Quality of Life - 6 Dimensions (AQoL-6D), a generic preference-based measure, is an appealing alternative to EQ-5D-5L for assessing health status in patients with chronic heart failure (HF), given its expanded scope. However, without a Malaysian value set, the AQoL-6D cannot generate health state utility values (HSUVs) to support local economic evaluations. This study intended to develop algorithms for predicting EQ-5D-5L HSUVs from AQoL-6D in an HF population.</p><p><strong>Methods: </strong>Cross-sectional data from a multicenter cohort of 419 HF outpatients were used. Both direct and indirect mapping approaches were attempted using 5 sets of explanatory variables and 8 models (ordinary least squares, Tobit, censored least absolute deviations, generalized linear model, 2-part model [TPM], beta regression-based model, adjusted limited dependent variable mixture model, and multinomial ordinal regression [MLOGIT]). The models' predictive performance was assessed through 10-fold cross-validated mean absolute error [MAE] and root mean squared error [RMSE]). Potential prediction bias was also examined graphically. The best-performing models, with the lowest RMSE and no bias, were then identified.</p><p><strong>Results: </strong>Among the models evaluated, TPM, which included age, sex, and 5 AQoL-6D dimension scores as predictors, appears to be the best-performing model for directly predicting EQ-5D-5L HSUVs from AQoL-6D. TPM yielded the lowest MAE (0.0802) and RMSE (0.1116), and demonstrated predictive accuracy for HSUVs >0.2 without significant bias. A MLOGIT model developed for response mapping had suboptimal predictive accuracy.</p><p><strong>Conclusions: </strong>This study developed potentially useful mapping algorithms for generating Malaysian EQ-5D-5L HSUVs from AQoL-6D responses among patients with HF when direct EQ-5D-5L data are unavailable.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914157","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cost-Effectiveness of Hepatocellular Carcinoma Surveillance Strategies in Patients With Compensated Liver Cirrhosis in the United Kingdom. 英国补偿性肝硬化患者 HCC 监控策略的成本效益。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-08 DOI: 10.1016/j.jval.2024.07.015
Osvaldo Ulises Garay, Louisa Elena Ambühl, Thomas G Bird, Eleanor Barnes, William L Irving, Ryan Walkley, Ian A Rowe

Objectives: This study aimed to evaluate the cost-effectiveness (CE) of 4 hepatocellular carcinoma (HCC) surveillance strategies in the United Kingdom, the GAAD algorithm, which combines Gender (biological sex) and Age with Elecsys® biomarker assays, alpha-fetoprotein (AFP) and protein induced by vitamin K absence-II (previously Des-γ-carboxy prothrombin); ultrasound (US); US + AFP and GAAD + US.

Methods: A de novo microsimulation state-transition Markov model was developed in Microsoft Excel® from the perspective of the United Kingdom National Health Service to calculate life-years, quality-adjusted life-years (QALYs), costs, incremental CE ratios, and net monetary benefits. Parameters were sourced from peer-reviewed published literature, national guidelines, and public cost databases. Sensitivity and scenario analyses were performed to evaluate the impact of parameter and structural uncertainty on the results.

Results: In a simulated cohort of 100 000 patients, discounted costs and QALYs per patient were £8663 and 6·066 for US, £9095 and 6·076 for US + AFP, £8719 and 6·078 for GAAD alone, and £9114 and 6·086 for GAAD + US. At a CE threshold of £20 000/QALY, GAAD was the most cost-effective strategy; however, although most costly, GAAD + US was the most clinically effective. Sensitivity and scenario analyses indicated that HCC incidence along with costs associated with diagnostic performance influence CE.

Conclusion: Considering the cost of US and low incidence of HCC in the United Kingdom, this study suggests that GAAD alone or in combination with US are cost-effective surveillance strategies compared with US and US + AFP. Although GAAD + US showed the highest QALY increase, GAAD alone is considered preferable regarding CE; however, better performance estimates for GAAD + US are needed to confirm.

研究目的本研究旨在评估英国四种肝细胞癌(HCC)监测策略的成本效益(CE),即GAAD算法(将性别(生物学性别)和年龄与Elecsys®生物标志物检测、甲胎蛋白(AFP)和维生素K缺失诱导蛋白-II(PIVKA-II)相结合)、超声波(US)、US+AFP和GAAD+US:方法:从英国国民健康服务的角度出发,在 Microsoft Excel® 中开发了一个全新的微观模拟状态转换马尔可夫模型,用于计算生命年、质量调整生命年 (QALY)、成本、增量 CE 比率和净货币收益。参数来源于同行评审发表的文献、国家指南和公共成本数据库。进行了敏感性和情景分析,以评估参数和结构不确定性对结果的影响:在 10 万名患者的模拟队列中,US 的每位患者贴现成本和 QALY 分别为 8,663 英镑和 6-066 英镑,US+AFP 分别为 9,095 英镑和 6-076,GAAD 单独为 8,719 英镑和 6-078,GAAD+US 分别为 9,114 英镑和 6-086。在 20,000 英镑/QALY 的 CE 临界值下,GAAD 是最具成本效益的策略;然而,尽管成本最高,但 GAAD+US 的临床效果最好。敏感性和情景分析表明,HCC发病率以及与诊断效果相关的成本会影响CE:考虑到 US 的成本和英国 HCC 的低发病率,本研究表明,与 US 和 US+AFP 相比,GAAD 单独使用或与 US 结合使用都是具有成本效益的监测策略。虽然 GAAD+US 的 QALY 增长率最高,但就 CE 而言,单独使用 GAAD 更为可取;不过,还需要对 GAAD+US 的性能进行更好的估算才能确认。
{"title":"Cost-Effectiveness of Hepatocellular Carcinoma Surveillance Strategies in Patients With Compensated Liver Cirrhosis in the United Kingdom.","authors":"Osvaldo Ulises Garay, Louisa Elena Ambühl, Thomas G Bird, Eleanor Barnes, William L Irving, Ryan Walkley, Ian A Rowe","doi":"10.1016/j.jval.2024.07.015","DOIUrl":"10.1016/j.jval.2024.07.015","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to evaluate the cost-effectiveness (CE) of 4 hepatocellular carcinoma (HCC) surveillance strategies in the United Kingdom, the GAAD algorithm, which combines Gender (biological sex) and Age with Elecsys® biomarker assays, alpha-fetoprotein (AFP) and protein induced by vitamin K absence-II (previously Des-γ-carboxy prothrombin); ultrasound (US); US + AFP and GAAD + US.</p><p><strong>Methods: </strong>A de novo microsimulation state-transition Markov model was developed in Microsoft Excel® from the perspective of the United Kingdom National Health Service to calculate life-years, quality-adjusted life-years (QALYs), costs, incremental CE ratios, and net monetary benefits. Parameters were sourced from peer-reviewed published literature, national guidelines, and public cost databases. Sensitivity and scenario analyses were performed to evaluate the impact of parameter and structural uncertainty on the results.</p><p><strong>Results: </strong>In a simulated cohort of 100 000 patients, discounted costs and QALYs per patient were £8663 and 6·066 for US, £9095 and 6·076 for US + AFP, £8719 and 6·078 for GAAD alone, and £9114 and 6·086 for GAAD + US. At a CE threshold of £20 000/QALY, GAAD was the most cost-effective strategy; however, although most costly, GAAD + US was the most clinically effective. Sensitivity and scenario analyses indicated that HCC incidence along with costs associated with diagnostic performance influence CE.</p><p><strong>Conclusion: </strong>Considering the cost of US and low incidence of HCC in the United Kingdom, this study suggests that GAAD alone or in combination with US are cost-effective surveillance strategies compared with US and US + AFP. Although GAAD + US showed the highest QALY increase, GAAD alone is considered preferable regarding CE; however, better performance estimates for GAAD + US are needed to confirm.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914154","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cost-Effectiveness of Axicabtagene Ciloleucel for Adult Patients With Relapsed or Refractory Follicular Lymphoma in the United States 美国治疗复发或难治性滤泡性淋巴瘤成人患者的阿昔单抗西乐葆的成本效益。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-01 DOI: 10.1016/j.jval.2024.04.003

Objectives

The results of a recent single-arm trial (ZUMA-5) of axicabtagene ciloleucel (axi-cel) for relapsed/refractory (r/r) follicular lymphoma (FL) demonstrated high rates of durable response and tolerable toxicity among treated patients. To quantify the value of axi-cel compared with standard of care (SOC) to manage r/r FL patients who have had at least 2 prior lines of systemic therapy (3L+), a cost-effectiveness model was developed from a US third-party payer perspective.

Methods

A 3-state partitioned-survival cost-effectiveness model was developed with a lifetime horizon. Patient-level analyses of the 36-month ZUMA-5 (axi-cel) and SCHOLAR-5 (SOC) studies were used to extrapolate progression-free and overall survivals. After 5 years of survival, an estimated 40% of the modeled population was assumed to experience long-term remission based on literature. Results include the incremental cost-effectiveness ratio (ICER) measured as incremental cost per quality-adjusted life year (QALY) gained. One-way sensitivity analysis, probabilistic sensitivity analysis, and scenario analyses were performed. All outcomes were discounted 3% per year.

Results

Axi-cel led to an increase of 4.28 life-years, 3.64 QALYs, and a total cost increase of $321 192 relative to SOC, resulting in an ICER of $88 300 per QALY. Across all parameters varied in the one-way sensitivity analysis, the ICER varied between $133 030 and $67 277. In the probabilistic sensitivity analysis, axi-cel had a 99% probability of being cost-effective across 5000 iterations using a $150 000 willingness-to-pay threshold.

Conclusions

Given the robustness of the model results and sensitivity analyses, axi-cel is expected to be a cost-effective treatment in 3L+ r/r FL.

目的最近进行的一项阿昔卡巴替林西洛昔洛韦(axicabtagene ciloleucel,axi-cel)治疗复发/难治性滤泡性淋巴瘤(FL)的单臂试验(ZUMA-5)结果显示,接受治疗的患者中持久应答率高且毒性可耐受。为了量化axi-cel与标准疗法(SOC)相比对既往接受过至少2种系统疗法(3L+)的复发性/难治性滤泡性淋巴瘤患者的治疗价值,我们从美国第三方支付机构的角度建立了一个成本效益模型。对 ZUMA-5(axi-cel)和 SCHOLAR-5(SOC)为期 36 个月的研究进行了患者层面的分析,以推断无进展生存期和总生存期。根据文献资料,在 5 年生存期后,估计有 40% 的建模人群会出现长期缓解。结果包括增量成本效益比 (ICER),即每获得质量调整生命年 (QALY) 的增量成本。进行了单向敏感性分析、概率敏感性分析和情景分析。结果相对于SOC,Axi-cel可增加4.28个生命年、3.64个质量调整生命年,总成本增加321 192美元,每质量调整生命年的ICER为88 300美元。在单向敏感性分析中,在所有参数变化的情况下,ICER 在 133 030 美元到 67 277 美元之间变化。结论鉴于模型结果和敏感性分析的稳健性,在3L+ r/r FL中,axi-cel有望成为一种具有成本效益的治疗方法。
{"title":"Cost-Effectiveness of Axicabtagene Ciloleucel for Adult Patients With Relapsed or Refractory Follicular Lymphoma in the United States","authors":"","doi":"10.1016/j.jval.2024.04.003","DOIUrl":"10.1016/j.jval.2024.04.003","url":null,"abstract":"<div><h3>Objectives</h3><p>The results of a recent single-arm trial (ZUMA-5) of axicabtagene ciloleucel<span> (axi-cel) for relapsed/refractory (r/r) follicular lymphoma (FL) demonstrated high rates of durable response and tolerable toxicity among treated patients. To quantify the value of axi-cel compared with standard of care (SOC) to manage r/r FL patients who have had at least 2 prior lines of systemic therapy (3L+), a cost-effectiveness model was developed from a US third-party payer perspective.</span></p></div><div><h3>Methods</h3><p>A 3-state partitioned-survival cost-effectiveness model was developed with a lifetime horizon. Patient-level analyses of the 36-month ZUMA-5 (axi-cel) and SCHOLAR-5 (SOC) studies were used to extrapolate progression-free and overall survivals. After 5 years of survival, an estimated 40% of the modeled population was assumed to experience long-term remission based on literature. Results include the incremental cost-effectiveness ratio (ICER) measured as incremental cost per quality-adjusted life year (QALY) gained. One-way sensitivity analysis, probabilistic sensitivity analysis, and scenario analyses were performed. All outcomes were discounted 3% per year.</p></div><div><h3>Results</h3><p>Axi-cel led to an increase of 4.28 life-years, 3.64 QALYs, and a total cost increase of $321 192 relative to SOC, resulting in an ICER of $88 300 per QALY. Across all parameters varied in the one-way sensitivity analysis, the ICER varied between $133 030 and $67 277. In the probabilistic sensitivity analysis, axi-cel had a 99% probability of being cost-effective across 5000 iterations using a $150 000 willingness-to-pay threshold.</p></div><div><h3>Conclusions</h3><p>Given the robustness of the model results and sensitivity analyses, axi-cel is expected to be a cost-effective treatment in 3L+ r/r FL.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140757067","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Long COVID Symptoms and Severity Score: Development, Validation, and Application 长冠状病毒病(COVID)症状和严重程度评分:开发、验证和应用。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-01 DOI: 10.1016/j.jval.2024.04.009

Objectives

The primary focus of this research is the proposition of a methodological framework for the clinical application of the long COVID symptoms and severity score (LC-SSS). This tool is not just a self-reported assessment instrument developed and validated but serves as a standardized, quantifiable means to monitor the diverse and persistent symptoms frequently observed in individuals with long COVID.

Methods

A 3-stage process was used to develop, validate, and establish scoring standards for the LC-SSS. Validation measures included correlations with other patient-reported measures, confirmatory factor analysis, Cronbach’s α for internal consistency, and test-retest reliability. Scoring standards were determined using K-means clustering, with comparative assessments made against hierarchical clustering and the Gaussian Mixture Model.

Results

The LC-SSS showed correlations with EuroQol 5-Dimension 5-Level (rs = −0.55), EuroQol visual analog scale (rs = −0.368), Patient Health Questionnaire-9 (rs = 0.538), Beck Anxiety Inventory (rs = 0.689), and Insomnia Severity Index (rs = 0.516), confirming its construct validity. Structural validity was good with a comparative fit index of 0.969, with Cronbach’s α of 0.93 indicating excellent internal consistency. Test-retest reliability was also satisfactory (intraclass correlation coefficient 0.732). K-means clustering identified 3 distinct severity categories in individuals living with long COVID, providing a basis for personalized treatment strategies.

Conclusions

The LC-SSS provides a robust and valid tool for assessing long COVID. The severity categories established via K-means clustering demonstrate significant variation in symptom severity, informing personalized treatment and improving care quality for patients with long COVID.

研究目的本研究的主要重点是为长COVID症状和严重程度评分(LC-SSS)的临床应用提出一个方法框架。该工具不仅是一种经过开发和验证的自我报告评估工具,而且还是一种标准化、可量化的手段,用于监测长 COVID 患者经常出现的各种持续性症状。研究方法通过三个阶段来开发、验证和建立 LC-SSS 的评分标准。验证措施包括与其他患者报告措施的相关性、确认性因素分析、内部一致性 Cronbach's α 和测试-再测可靠性。结果 LC-SSS 与 EuroQol 5-Dimension 5-Level 相关(rs = -0.55)、EuroQol 视觉模拟量表(rs = -0.368)、患者健康问卷-9(rs = 0.538)、贝克焦虑量表(rs = 0.689)和失眠严重程度指数(rs = 0.516)相关,证实了其结构效度。结构效度良好,比较拟合指数为 0.969,Cronbach's α 为 0.93,表明内部一致性极佳。重测可靠性也令人满意(类内相关系数为 0.732)。K-means 聚类确定了长时 COVID 患者的 3 个不同严重程度类别,为个性化治疗策略提供了依据。通过 K-means 聚类确定的严重程度类别显示了症状严重程度的显著差异,为个性化治疗提供了依据,并提高了长程 COVID 患者的护理质量。
{"title":"The Long COVID Symptoms and Severity Score: Development, Validation, and Application","authors":"","doi":"10.1016/j.jval.2024.04.009","DOIUrl":"10.1016/j.jval.2024.04.009","url":null,"abstract":"<div><h3>Objectives</h3><p>The primary focus of this research is the proposition of a methodological framework for the clinical application of the long COVID symptoms and severity score (LC-SSS). This tool is not just a self-reported assessment instrument developed and validated but serves as a standardized, quantifiable means to monitor the diverse and persistent symptoms frequently observed in individuals with long COVID.</p></div><div><h3>Methods</h3><p>A 3-stage process was used to develop, validate, and establish scoring standards for the LC-SSS. Validation measures included correlations with other patient-reported measures, confirmatory factor analysis, Cronbach’s α for internal consistency, and test-retest reliability. Scoring standards were determined using K-means clustering, with comparative assessments made against hierarchical clustering and the Gaussian Mixture Model.</p></div><div><h3>Results</h3><p>The LC-SSS showed correlations with EuroQol 5-Dimension 5-Level (r<sub>s</sub> = −0.55), EuroQol visual analog scale (r<sub>s</sub> = −0.368), Patient Health Questionnaire-9 (r<sub>s</sub> = 0.538), Beck Anxiety Inventory (r<sub>s</sub> = 0.689), and Insomnia Severity Index (r<sub>s</sub> = 0.516), confirming its construct validity. Structural validity was good with a comparative fit index of 0.969, with Cronbach’s α of 0.93 indicating excellent internal consistency. Test-retest reliability was also satisfactory (intraclass correlation coefficient 0.732). K-means clustering identified 3 distinct severity categories in individuals living with long COVID, providing a basis for personalized treatment strategies.</p></div><div><h3>Conclusions</h3><p>The LC-SSS provides a robust and valid tool for assessing long COVID. The severity categories established via K-means clustering demonstrate significant variation in symptom severity, informing personalized treatment and improving care quality for patients with long COVID.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524023416/pdfft?md5=86160a63dcd23c8386405b2f4c4a4dfc&pid=1-s2.0-S1098301524023416-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140791012","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
How Do the Institute for Clinical and Economic Review’s Assessments of Comparative Effectiveness Compare With the German Federal Joint Committee’s Assessments of Added Benefit? A Qualitative Study 临床与经济审查研究所的比较效益评估与德国联邦联合委员会的附加效益评估相比如何?定性研究。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-01 DOI: 10.1016/j.jval.2024.04.015

Objectives

We compared the Institute for Clinical and Economic Review’s (ICER) ratings of comparative clinical effectiveness with the German Federal Joint Committee’s (G-BA) added benefit ratings, and explored what factors may explain the disagreement between the 2 organizations.

Methods

We included drugs if they were assessed by ICER under its 2020 to 2023 Value Assessment Framework and had a corresponding assessment by G-BA as of January 2024 for the same indication, patient population, and comparator drug. To compare assessments, we modified ICER’s proposed crosswalk between G-BA and ICER benefit ratings to account for G-BA’s certainty ratings. We also determined whether each pair was based on similar evidence. Assessment pairs exhibiting disagreement based on the modified crosswalk despite a similar evidence base were qualitatively analyzed to identify reasons for disagreement.

Results

Out of 15 drug assessment pairs matched on indication, patient subgroup, and comparator, none showed agreement in their assessments when based on similar evidence. Disagreement was attributed to differences in evidence evaluation, including evaluations of safety, generalizability, and study design, as well as G-BA’s rejection of the available evidence in 4 cases as unsuitable.

Conclusions

The findings demonstrate that even under conditions where populations and comparators are identical and the evidence base is consistent, different assessors may arrive at divergent conclusions about comparative effectiveness, thus underscoring the presence of value judgments within assessments of clinical effectiveness. To support initiatives that seek to facilitate the exchange of value assessments between countries, these value judgments should always be transparently presented and justified in assessment summaries.

目的:我们比较了美国临床与经济研究所(ICER)的比较临床有效性评级和德国联邦联合委员会(G-BA)的附加效益评级,并探讨了哪些因素可能导致两个机构之间出现分歧:如果 ICER 在其 2020-2023 年价值评估框架下对药物进行了评估,且 G-BA 在 2024 年 1 月对相同适应症、患者人群和对比药物进行了相应评估,则我们将这些药物纳入评估范围。为了比较评估结果,我们修改了 ICER 提出的 G-BA 和 ICER 效益评级之间的交叉关系,以考虑 G-BA 的确定性评级。我们还确定了每对评估是否基于相似的证据。尽管有相似的证据基础,但根据修改后的交叉线对出现分歧的评估对进行了定性分析,以确定出现分歧的原因:结果:在根据适应症、患者亚群和参照物进行匹配的 15 对药物评估中,没有一对在基于相似证据的评估中表现出一致。分歧归因于证据评估的不同,包括对安全性、可推广性和研究设计的评估,以及G-BA在四个案例中认为现有证据不合适而拒绝接受:研究结果表明,即使在人群和比较对象相同、证据基础一致的情况下,不同的评估者也可能得出不同的比较效果结论,从而强调了临床效果评估中价值判断的存在。为了支持旨在促进国家间价值评估交流的倡议,这些价值判断应始终在评估摘要中以透明的方式呈现并说明理由。
{"title":"How Do the Institute for Clinical and Economic Review’s Assessments of Comparative Effectiveness Compare With the German Federal Joint Committee’s Assessments of Added Benefit? A Qualitative Study","authors":"","doi":"10.1016/j.jval.2024.04.015","DOIUrl":"10.1016/j.jval.2024.04.015","url":null,"abstract":"<div><h3>Objectives</h3><p>We compared the Institute for Clinical and Economic Review’s (ICER) ratings of comparative clinical effectiveness with the German Federal Joint Committee’s (G-BA) added benefit ratings, and explored what factors may explain the disagreement between the 2 organizations.</p></div><div><h3>Methods</h3><p>We included drugs if they were assessed by ICER under its 2020 to 2023 Value Assessment Framework and had a corresponding assessment by G-BA as of January 2024 for the same indication, patient population, and comparator drug. To compare assessments, we modified ICER’s proposed crosswalk between G-BA and ICER benefit ratings to account for G-BA’s certainty ratings. We also determined whether each pair was based on similar evidence. Assessment pairs exhibiting disagreement based on the modified crosswalk despite a similar evidence base were qualitatively analyzed to identify reasons for disagreement.</p></div><div><h3>Results</h3><p>Out of 15 drug assessment pairs matched on indication, patient subgroup, and comparator, none showed agreement in their assessments when based on similar evidence. Disagreement was attributed to differences in evidence evaluation, including evaluations of safety, generalizability, and study design, as well as G-BA’s rejection of the available evidence in 4 cases as unsuitable.</p></div><div><h3>Conclusions</h3><p>The findings demonstrate that even under conditions where populations and comparators are identical and the evidence base is consistent, different assessors may arrive at divergent conclusions about comparative effectiveness, thus underscoring the presence of value judgments within assessments of clinical effectiveness. To support initiatives that seek to facilitate the exchange of value assessments between countries, these value judgments should always be transparently presented and justified in assessment summaries.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140860425","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Estimating the Allocation of the Economic Value Generated by Utilization of All-Oral Direct-Acting Antivirals for Hepatitis C in the United States, 2015 to 2019 估算 2015-2019 年美国使用全口服直接作用抗病毒药物治疗丙型肝炎所产生的经济价值分配。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-01 DOI: 10.1016/j.jval.2024.04.011

Objectives

Between 2013 to 2019, several all-oral direct-acting antivirals (DAAs) were launched with the potential to cure patients with hepatitis C virus (HCV). They generated economic value in terms of the health gains for patients and cost-savings for the US healthcare system. We estimated the share of this value allocated to 4 manufacturers vs society.

Methods

For 2015 to 2019, we estimated the incremental impact of DAAs on HCV health outcomes and costs. We used the Center for Disease Analysis Foundation Polaris Observatory database to estimate utilization. Per-patient projections of lifetime quality-adjusted life-years (QALYs) gained and medical costs avoided were based on a standard 9-state HCV disease-progression model for DAA treatment vs alternatives. Annual QALY gains were valued at $114 000 per QALY. Outcomes and costs were discounted at 3%. Estimated revenues were based on reported sales.

Results

An estimated 1 080 000 patients received DAAs: 81.5% would not have received the pre-DAA standard of care. On average, these patients were projected to gain 4.4 QALYs and save $104 400 in lifetime healthcare costs, generating $531.8 billion in value. Those who would have received treatment gained 1.7 QALYs and saved $41 500 in lifetime costs, generating $47.4 billion in economic value. As treatment costs fell nearly 75%, the 4 manufacturers reported $37.4 billion from DAA sales—an allocation of 6.5% of the total value.

Conclusions

The significant majority (∼90%) of the economic value of curing HCV with DAAs were health benefits to patients and net cost-savings to society. DAA manufacturers received a minority share (6.5%) of the aggregate economic value generated.

目标从 2013 年到 2019 年,有几种全口服直接作用抗病毒药物(DAAs)上市,有望治愈丙型肝炎病毒(HCV)患者。这些药物为患者带来了健康收益,为美国医疗保健系统节省了成本,从而产生了经济价值。我们估算了这一价值分配给 4 家制造商与社会的份额。方法在 2015 年至 2019 年期间,我们估算了 DAAs 对 HCV 健康结果和成本的增量影响。我们使用疾病分析中心基金会 Polaris Observatory 数据库来估算使用情况。根据 DAA 治疗与替代治疗的标准 9 状态 HCV 疾病进展模型,预测了每位患者终生获得的质量调整生命年 (QALY) 和避免的医疗费用。每年获得的 QALY 按每 QALY 114 000 美元估价。结果和成本的贴现率为 3%。估计收入基于报告的销售额:81.5%的患者不会接受DAA前的标准治疗。平均而言,这些患者预计将获得 4.4 QALYs,并在终身医疗保健成本方面节省 104 400 美元,产生 5,318 亿美元的价值。本应接受治疗的患者则可获得 1.7 QALYs,终生节省成本 41500 美元,产生 474 亿美元的经济价值。由于治疗成本下降了近 75%,4 家生产商报告的 DAA 销售额为 374 亿美元,占总价值的 6.5%。DAA生产商只获得了总经济价值的少数份额(6.5%)。
{"title":"Estimating the Allocation of the Economic Value Generated by Utilization of All-Oral Direct-Acting Antivirals for Hepatitis C in the United States, 2015 to 2019","authors":"","doi":"10.1016/j.jval.2024.04.011","DOIUrl":"10.1016/j.jval.2024.04.011","url":null,"abstract":"<div><h3>Objectives</h3><p>Between 2013 to 2019, several all-oral direct-acting antivirals (DAAs) were launched with the potential to cure patients with hepatitis C virus (HCV). They generated economic value in terms of the health gains for patients and cost-savings for the US healthcare system. We estimated the share of this value allocated to 4 manufacturers vs society.</p></div><div><h3>Methods</h3><p>For 2015 to 2019, we estimated the incremental impact of DAAs on HCV health outcomes and costs. We used the Center for Disease Analysis Foundation Polaris Observatory database to estimate utilization. Per-patient projections of lifetime quality-adjusted life-years (QALYs) gained and medical costs avoided were based on a standard 9-state HCV disease-progression model for DAA treatment vs alternatives. Annual QALY gains were valued at $114 000 per QALY. Outcomes and costs were discounted at 3%. Estimated revenues were based on reported sales.</p></div><div><h3>Results</h3><p>An estimated 1 080 000 patients received DAAs: 81.5% would not have received the pre-DAA standard of care. On average, these patients were projected to gain 4.4 QALYs and save $104 400 in lifetime healthcare costs, generating $531.8 billion in value. Those who would have received treatment gained 1.7 QALYs and saved $41 500 in lifetime costs, generating $47.4 billion in economic value. As treatment costs fell nearly 75%, the 4 manufacturers reported $37.4 billion from DAA sales—an allocation of 6.5% of the total value.</p></div><div><h3>Conclusions</h3><p>The significant majority (∼90%) of the economic value of curing HCV with DAAs were health benefits to patients and net cost-savings to society. DAA manufacturers received a minority share (6.5%) of the aggregate economic value generated.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S109830152402343X/pdfft?md5=30bfecad282596af1a8bdaadf2258d75&pid=1-s2.0-S109830152402343X-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140778379","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Health Fluctuations in Dementia and its Impact on the Assessment of Health-Related Quality of Life Using the EQ-5D-5L 痴呆症患者的健康波动及其对使用 EQ-5D-5L 评估健康相关生活质量的影响。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-01 DOI: 10.1016/j.jval.2024.04.010

Objectives

To quantify health fluctuations, identify affected health-related quality of life (HRQoL) dimensions, and evaluate if fluctuations affect the HRQoL instruments recall period adherence in people living with dementia (PlwD).

Methods

Caregivers of PlwD completed a daily diary for 14 days, documenting if PlwD’s health was better or worse than the day before and the affected HRQoL dimensions. Health fluctuation was categorized into low (0-4 fluctuations in 14 days), moderate (5-8), and high (9-14). Also, caregivers and PlwD completed the EQ-5D-5L (proxy- and self-reported) on days 1, 7, and 14. Subsequently, caregivers were interviewed to determine whether recurrent fluctuations were considered in the EQ-5D-5L assessment of today’s health (recall period adherence).

Results

Fluctuations were reported for 96% of PlwD, on average, for 7 of the 14 days. Dimensions most frequently triggering fluctuations included memory, mobility, concentration, sleep, pain, and usual activities. Fluctuations were associated with higher EQ-5D-5L health-states variation and nonadherence to the EQ-5D-5L recall period “today.” PlwD with moderate to high fluctuation had the highest EQ-5D-5L utility change between day 1 and 14 (0.157 and 0.134) and recall period nonadherence (31% and 26%) compared with PlwD with low fluctuation (0.010; 17%). Recall period nonadherence was higher in PlwD with improved compared with those with deteriorated health in the diary (37% vs 9%).

Conclusions

Health fluctuations frequently occur in dementia and strongly affect HRQoL assessments. Further research is needed to evaluate if more extended recall periods and multiple, consecutive assessments could capture health fluctuations more appropriately in dementia.

方法痴呆症患者(PlwD)的护理人员填写为期 14 天的每日日记,记录痴呆症患者的健康状况与前一天相比是好是坏以及受影响的 HRQoL 维度。健康波动分为低度(14 天内波动 0-4 次)、中度(5-8 次)和高度(9-14 次)。此外,护理人员和 PlwD 在第 1、7 和 14 天填写了 EQ-5D-5L(代理和自我报告)。随后,护理人员接受了访谈,以确定在 EQ-5D-5L 评估当今健康状况时是否考虑了反复波动(回忆期坚持)。最常引发波动的因素包括记忆力、活动能力、注意力、睡眠、疼痛和日常活动。波动与较高的 EQ-5D-5L 健康状态变化和不遵守 EQ-5D-5L "今天 "回忆期有关。与波动性低的 PlwD(0.010;17%)相比,波动性中高的 PlwD 在第 1 天和第 14 天之间的 EQ-5D-5L 效用变化(0.157 和 0.134)和回顾期不坚持率(31% 和 26%)最高。与日记中健康状况恶化的患者相比,健康状况改善的 PlwD 患者在回忆期间不坚持治疗的比例更高(37% vs 9%)。还需要进一步研究,以评估更长的回忆期和多次连续评估是否能更恰当地捕捉到痴呆症患者的健康波动。
{"title":"Health Fluctuations in Dementia and its Impact on the Assessment of Health-Related Quality of Life Using the EQ-5D-5L","authors":"","doi":"10.1016/j.jval.2024.04.010","DOIUrl":"10.1016/j.jval.2024.04.010","url":null,"abstract":"<div><h3>Objectives</h3><p>To quantify health fluctuations, identify affected health-related quality of life (HRQoL) dimensions, and evaluate if fluctuations affect the HRQoL instruments recall period adherence in people living with dementia (PlwD).</p></div><div><h3>Methods</h3><p>Caregivers of PlwD completed a daily diary for 14 days, documenting if PlwD’s health was better or worse than the day before and the affected HRQoL dimensions. Health fluctuation was categorized into low (0-4 fluctuations in 14 days), moderate (5-8), and high (9-14). Also, caregivers and PlwD completed the EQ-5D-5L (proxy- and self-reported) on days 1, 7, and 14. Subsequently, caregivers were interviewed to determine whether recurrent fluctuations were considered in the EQ-5D-5L assessment of today’s health (recall period adherence).</p></div><div><h3>Results</h3><p>Fluctuations were reported for 96% of PlwD, on average, for 7 of the 14 days. Dimensions most frequently triggering fluctuations included memory, mobility, concentration, sleep, pain, and usual activities. Fluctuations were associated with higher EQ-5D-5L health-states variation and nonadherence to the EQ-5D-5L recall period “today.” PlwD with moderate to high fluctuation had the highest EQ-5D-5L utility change between day 1 and 14 (0.157 and 0.134) and recall period nonadherence (31% and 26%) compared with PlwD with low fluctuation (0.010; 17%). Recall period nonadherence was higher in PlwD with improved compared with those with deteriorated health in the diary (37% vs 9%).</p></div><div><h3>Conclusions</h3><p>Health fluctuations frequently occur in dementia and strongly affect HRQoL assessments. Further research is needed to evaluate if more extended recall periods and multiple, consecutive assessments could capture health fluctuations more appropriately in dementia.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524023428/pdfft?md5=b32743f1c93ee5cfb36816ffebf1bf17&pid=1-s2.0-S1098301524023428-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140770373","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
期刊
Value in Health
全部 Acc. Chem. Res. ACS Applied Bio Materials ACS Appl. Electron. Mater. ACS Appl. Energy Mater. ACS Appl. Mater. Interfaces ACS Appl. Nano Mater. ACS Appl. Polym. Mater. ACS BIOMATER-SCI ENG ACS Catal. ACS Cent. Sci. ACS Chem. Biol. ACS Chemical Health & Safety ACS Chem. Neurosci. ACS Comb. Sci. ACS Earth Space Chem. ACS Energy Lett. ACS Infect. Dis. ACS Macro Lett. ACS Mater. Lett. ACS Med. Chem. Lett. ACS Nano ACS Omega ACS Photonics ACS Sens. ACS Sustainable Chem. Eng. ACS Synth. Biol. Anal. Chem. BIOCHEMISTRY-US Bioconjugate Chem. BIOMACROMOLECULES Chem. Res. Toxicol. Chem. Rev. Chem. Mater. CRYST GROWTH DES ENERG FUEL Environ. Sci. Technol. Environ. Sci. Technol. Lett. Eur. J. Inorg. Chem. IND ENG CHEM RES Inorg. Chem. J. Agric. Food. Chem. J. Chem. Eng. Data J. Chem. Educ. J. Chem. Inf. Model. J. Chem. Theory Comput. J. Med. Chem. J. Nat. Prod. J PROTEOME RES J. Am. Chem. Soc. LANGMUIR MACROMOLECULES Mol. Pharmaceutics Nano Lett. Org. Lett. ORG PROCESS RES DEV ORGANOMETALLICS J. Org. Chem. J. Phys. Chem. J. Phys. Chem. A J. Phys. Chem. B J. Phys. Chem. C J. Phys. Chem. Lett. Analyst Anal. Methods Biomater. Sci. Catal. Sci. Technol. Chem. Commun. Chem. Soc. Rev. CHEM EDUC RES PRACT CRYSTENGCOMM Dalton Trans. Energy Environ. Sci. ENVIRON SCI-NANO ENVIRON SCI-PROC IMP ENVIRON SCI-WAT RES Faraday Discuss. Food Funct. Green Chem. Inorg. Chem. Front. Integr. Biol. J. Anal. At. Spectrom. J. Mater. Chem. A J. Mater. Chem. B J. Mater. Chem. C Lab Chip Mater. Chem. Front. Mater. Horiz. MEDCHEMCOMM Metallomics Mol. Biosyst. Mol. Syst. Des. Eng. Nanoscale Nanoscale Horiz. Nat. Prod. Rep. New J. Chem. Org. Biomol. Chem. Org. Chem. Front. PHOTOCH PHOTOBIO SCI PCCP Polym. Chem.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1